Aeterna Zentaris Inc., a late-stage oncology drug development company, announced that its Japanese partner, Yakult Honsha (Yakult), has initiated a phase I/II trial in Japan to assess the safety and efficacy of the company’s PI3K/Akt inhibitor, perifosine, in combination with chemotherapeutic agent, capecitabine, in patients with refractory advanced colorectal cancer (CRC).

The initiation of this trial will trigger a milestone payment of an undisclosed amount from Yakult to Aeterna Zentaris under the partnership agreement signed with Yakult in March 2011 for perifosine in Japan.

Juergen Engel, PhD, president and CEO of Aeterna Zentaris stated, “We would like to thank our partner Yakult for the swift initiation of this trial in Japan, a first in the clinical development of perifosine in Japan. The trial is also part of our worldwide clinical development and registration strategy for perifosine in colorectal cancer, as we await the completion of the phase III trial in this indication in the United States during the first quarter of 2012.”

The phase I/II trial is being conducted in patients with refractory advanced CRC. The primary endpoint of the phase 1 portion of the trial is the safety profile of perifosine in combination with capecitabine. The primary endpoint of the phase II portion is efficacy (Disease Control Rate).

Perifosine is a novel, oral anticancer treatment that inhibits Akt activation in the phosphoinositide 3-kinase (PI3K) pathway. The product works by interfering with membranes of cancer cells thereby inhibiting Akt signalling which then affects cell death, growth, differentiation and survival. Perifosine, in combination with chemotherapeutic agents, is currently being studied for the treatment of colorectal cancer, multiple myeloma and other cancers, and is the most advanced anticancer agent of its class.

Perifosine, as monotherapy, is being explored in other indications. The FDA has granted perifosine orphan drug designation in multiple myeloma and neuroblastoma, and Fast Track designations in both multiple myeloma and refractory advanced colorectal cancer. Additionally, an agreement was reached with the FDA to conduct the phase III trials in both of these indications under a Special Protocol Assessment.

Perifosine has also been granted orphan medicinal product designation from the European Medicines Agency (EMA) in multiple myeloma. Furthermore, perifosine has received positive Scientific Advice from the EMA for both the multiple myeloma and advanced colorectal cancer programs, with ongoing phase III trials for these indications expected to be sufficient for registration in Europe. Perifosine rights have been licensed to Keryx Biopharmaceuticals, Inc. for North America, to Yakult for Japan, to Handok for Korea and to Hikma Pharmaceuticals for the MENA (Middle East and North Africa) region.

Yakult is focused on the development and marketing of pharmaceuticals, foods, beverages, and cosmetics.

Aeterna Zentaris is a late-stage oncology drug development company currently investigating potential treatments for various cancers including colorectal, multiple myeloma, endometrial, ovarian, prostate and bladder cancer.